ViaCyte starts clinical trial on PEC-Direct islet cell replacement therapy

ViaCyte is a regenerative medicine company, and they have recently announced that an exciting clinical trial, partially funded by JDRF, is underway. 

The first patients have already been implanted with PEC-Direct product candidate.  This is a novel islet cell replacement therapy that they are developing.  It would be a long-term treatment for Type 1 diabetes patients who have a high likelihood of developing acute life-threatening complications of the disease. 

These complications include:

• hypoglycemia unawareness
• labile blood glucose (BG) levels
• episodes of severe hypoglycemia.

ViaCyte’s product candidates use stem cells to derive pancreatic progenitor cells, which are implanted in a durable and retrievable cell delivery device.  After the cells are implanted and reach maturity, they make insulin and other pancreatic hormones based upon blood glucose levels.  The PEC-Direct product candidate uses a non-immunoprotective device to deliver the pancreatic progenitor cells and is intended for high-risk patients with Type 1 diabetes.  They are also developing another product candidate called PEC-Encap, which uses an immunoprotective device to deliver the same pancreatic progenitor cells.  This is in development for all insulin-treated patients, including those with Type 2.

How it works:

In the clinical trial, the first group of patients with Type 1 have been implanted with sentinels, which are multiple small-format cell-filled devices, with the goal of evaluation the safety and viability of the implants.  The sentinels will be removed at certain points in time, and evaluated at the tissue level to gain insight into the process of the cells engrafting and maturing into pancreatic islet cells, including insulin-producing beta cells.  Later this year, it is anticipated that a second group of no more than 40 patients will begin enrollment, with the goal of evaluating safety and efficacy.  The primary determinant of efficacy will be the clinically relevant production of insulin, as measured by C-Peptide levels. 

This would be very exciting to see in a population of patients who, at enrollment, have little to no ability to produce endogenous insulin.  Researchers will evaluate other important endpoints such as use of injected insulin and the incidence of hypoglycemia.  ViaCyte hopes to show preliminary evidence of efficacy in the first six months of 2018, and definitive efficacy by no later than mid-2018.

The Bottom Line:

Past research has shown success in treating patients with unstable, high risk type 1 diabetes with islet transplants.  But there is a limited supply of donor organs, and it’s difficult to source consistent and reliable islet preparations.  Islet replacement therapy sourced from stem cells would circumvent these problems and may be able to help a larger number of patients.  PEC-Direct islet cell replacement therapy can help those who have the greatest medical need.

It’s exciting to hear about what is in development to provide better treatments and improved quality of life for people living with diabetes!